Alnylam 5×15

Our “Alnylam 5×15″ and genetic medicine strategy, launched in January 2011, establishes a path for development and commercialization of novel RNAi therapeutics toward genetically defined targets for the treatment of diseases with high unmet medical need.

Products arising from this strategy share several critical characteristics including:

  • the potential to have a major impact in a high unmet need population;
  • a genetically defined, liver-expressed target gene involved in disease and validated in human genetics;
  • the ability to leverage the existing Alnylam RNAi delivery platform;
  • the opportunity to monitor an early, blood-based biomarker with strong disease correlation in Phase 1 clinical trials for human proof of concept; and,
  • the existence of clinically relevant endpoints for the filing of a new drug application (NDA) with a focused patient database and possible accelerated paths for commercialization. 

As recently updated in early 2014, we expect to have six to seven RNAi therapeutic programs in clinical development, including two programs in Phase 3, and five to six programs with human proof of concept by the end of 2015.  Our “Alnylam 5×15″ and genetic medicine programs in development include:

  • Patisiran (ALN-TTR02) targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR) in patients with familial amyloidotic polyneuropathy (FAP)
  • ALN-TTRsc targeting TTR for the treatment of ATTR in patients with familial amyloidotic cardiomyopathy (FAC)
  • ALN-AT3 targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD)
  • ALN-CC5 targeting complement component C5 for the treatment of complement-mediated disease
  • ALN-AS1 targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of porphyria including acute intermittent porphyria (AIP)
  • ALN-AAT targeting alpha-1-antitrypsin (AAT) for the treatment of AAT deficiency liver disease
  • ALN-TMP targeting TMPRSS6 for the treatment of beta-thalassemia and iron-overload disorders
  • ALN-PCS targeting PCSK9 for the treatment of hypercholesterolemia
  • ALN-ANG targeting ANGPTL3 for the treatment of genetic forms of mixed hyperlipidemia and severe hypertriglyceridemia
  • Other programs.